If a drug was developed to block a key protein linked to the onset of Huntington's Disease, it could have a clear path to the part of the brain most affected by the disease, while not bothering other parts of the brain and body, said distinguished neuroscientist Solomon H. Snyder, MD, to an audience of about 300 students and faculty at the University of Maryland, Baltimore (UMB).
Snyder said, "Even though there is a market out there of only 100,000 patients," his laboratory at Johns Hopkins University is currently partnering with big pharmaceutical companies to develop such a drug, one to block binding of the Rhes protein to mutant huntingtin protein, a genetically altered protein in Huntington's Disease which kills cells in the brain's corpus striatum.
Snyder presented the annual Ellis S. Grollman Lecture as the guest of the School of Pharmacy at UMB. His scientific publications have had vast impact; thus he is the world's most cited biomedical researcher.
He began the lecture by "walking" the students, he said, through the laborious and sometimes frustrating pathway to scientific discovery. He explained results of many "great papers in good journals" since 1975 from his and other laboratories that provided insights into the biochemistry of Huntington's. But each paper failed to pinpoint why the disease shrinks the brain's corpus striatum drastically and very selectively. "It was still a big mystery, though we do know that it is caused by a single gene mutation," said Snyder.
Huntington's disease is caused by a mutation in the protein huntingtin (Htt) that is expressed throughout the body and all the brain regions. But the pathology of Huntington's is mostly confined to the corpus striatum, said Snyder.
The Ellis Grollman Lecture in the Pharmaceutical Sciences was established at the School by his sister, Mr. Evelyn Grollman Glick, as an endowed memorial. Natalie D. Eddington, PhD, dean of the School of Pharmacy said that the annual lecture focuses on distinguished biomedical research with an emphasis on biological systems.
"It was an honor for the Department of Pharmaceutical Sciences and School of Pharmacy to host Dr. Snyder," said Andrew Coop, PhD, professor and chair of the department. "The impact of his groundbreaking research in the area of neuroscience cannot be overstated, and has led to major advances in the biomedical sciences directly improving public health."
Small brain-penetrating molecule offers hope for treating aggressive brain tumors